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Five Years of Patient Registry Success: NORD’s IAMRARE™ Registry Program Celebrates New Partnerships and Models of Engagement  

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Washington, D.C., October 3, 2018—The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans with rare diseases, today is celebrating five years of patient registry success with the announcement of a new multi-stakeholder model of engagement in collaboration with the Foundation for Prader-Willi Research and Zafgen, Inc.


In the rare disease space, with relatively small patient populations, the power of data is diluted when split across multiple registry platforms. In order to reduce duplicative registry efforts, NORD has developed a solution to address the needs across stakeholder groups whereby researchers from industry, academia, and medicine can establish sub-studies to capture distinct data related to their project needs against the full registry population, or study specific trends in a sub-set of the patient population. This new functionality allows for partnerships that bring stakeholders and experts together to accelerate the pace of research for rare conditions, while ensuring patient communities remain empowered as partners throughout the process. This expanded functionality provides a direct pathway for industry and researchers to partner with NORD and patient organizations.


The first sub-study model on NORD’s platform includes a partnership between Zafgen and the Foundation for Prader-Willi Research to capture data on a complex genetic condition characterized early on by weak muscle tone, feeding difficulties, poor growth, and developmental delay, and later by unrelenting hunger (hyperphagia), as well as slowed metabolism, behavioral and mental health challenges, and other co-morbidities. The PATH for PWS study aims to prospectively evaluate the incidence of serious medical events in participants with Prader-Willi syndrome (“PWS”) while retrospectively analyzing medical information to evaluate the natural history of PWS.


In a new video released by NORD, Janet Woodcock, Director of the Center for Drug Evaluation and Research at the Food and Drug Administration (FDA), echoes the need for such models of engagement and support for NORD’s registry program. The video can be viewed here.


By building a registry platform in close collaboration with the FDA, NORD has developed a user-friendly tool for collecting data urgently needed to better understand rare diseases and patient experiences, and ensure new treatment options become available. With thirty-two registry partnerships, nearly 7,000 active participants, and five years of robust data collection for a number of rare conditions, NORD remains committed to eliminating the barriers that prevent research into treatments and cures for the rare diseases that are without one, setting the stage for an open NORD platform that supports rare disease registries for all.


“NORD is proud to provide this vital and enduring resource for the rare disease community,” said Peter L. Saltonstall, President and CEO of NORD. “With 95% of rare diseases still without an FDA-approved treatment or cure, we remain committed to eliminating hurdles that prevent research into rare diseases. We believe our platform and collaborative registry model has the power to do just that.”